Letter From The CEO
Dear Fellow Shareholders,
We received numerous calls and emails concerning Biogen-Idec’s recent announcement of clinical trial results demonstrating that the experimental drug, dexpramipexole, did not significantly alter disease progression or extend the life of patients with ALS. These results are, indeed, disappointing to all patients, friends and family directly affected by this devastating disease as well as those involved in the care and treatment of ALS patients. Despite years of research, millions of dollars spent and tremendous efforts by many to make new treatment options available, ALS remains a disease that claims too many lives each year.
In this letter I hope to clarify the difference of therapeutic approach between dexpramipexole and our ALS program.
Biogen-Idec licensed dexpramipexole from Knopp Biosciences in 2010 for development as a new treatment for ALS. It is a synthetic chemical, small molecule drug that has a number of significant differences from the therapeutic approach represented by our collaboration with Biogen-Idec. Dexpramipexole is a molecule that is thought to enhance nerve cell mitochondrial function (the internal power source for cellular life and activity) and protect neurons under stressors like those induced by ALS. This drug is required to penetrate neurons and the mitochondria in order to have this therapeutic effect.
The Amorfix Biogen-Idec approach is to develop an antibody therapeutic that directly neutralizes the neuron toxicity of misfolded superoxide-1 (SOD1). SOD1 in its native, properly folded form is a molecule that has many functions, one being to protect cells from the adverse effects of oxidants and free radicals that can kill neurons. In its misfolded form, SOD1 has the exact opposite effect in that it now facilitates the death of neurons rather than protect. Our antibodies are directed against misfolded SOD1 and, therefore, neutralize the adverse effects of this molecule while sparing natively folded SOD1 allowing it to remain as a protector of neurons and neuronal function.
Amorfix’s collaboration with Biogen-Idec and our therapeutic antibody program continue to move forward. Despite the tremendous disappointment from dexpramipexole results, Biogen-Idec has publicly stated that they will continue their work to investigate new treatments and therapeutic options for patients with ALS. We are privileged to be working with a company like Biogen-Idec that is as dedicated as we are to developing new treatments that will significantly improve the life and life span of individuals who suffer from ALS.
Robert Gundel, PhD, MBA
January 5, 2013